Seizure disorder drug developer Marinus Pharmaceuticals now has its first FDA approval, a decision that also marks the first approved treatment for a rare, inherited form of epilepsy.
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The FDA on Friday gave the regulatory green light to the drug, ganaxolone, as a treatment for cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder in patients age two and older. Radnor, Pennsylvania-based Marinus will market its new product under the name Ztalmy.
Patients who have CDKL5 deficiency experience difficult-to-control seizures and severe impairment of neurological development. The disease stems from a mutation of the CDKL5 gene, which encodes proteins that are key to normal development and function of the brain. This gene is located on the X chromosome; CDKL5 deficiency disproportionately affects females. Children who have the disease have problems walking, talking, and caring for themselves. Marinus estimates 12,500 patients in the U.S. and Europe have CDKL5 deficiency.
Ztalmy is a small molecule designed to bind to and activate type A GABA receptors in the central nervous system. This approach is intended to provide anticonvulsant effects. The Marinus drug is formulated as an oral suspension taken three times daily with food.
Marinus evaluated Ztalmy in a clinical trial enrolling 101 patients age two and older with confirmed CDKL5 genetic mutations, as well as seizures that are not adequately controlled by at least two prior therapies. Nearly 80% of trial participants were female, consistent with the demographics of the disease. The main goal of the double-blind and placebo-controlled Phase 3 study was to measure for the reduction in seizure frequency over a 17-week treatment period. The results showed that those treated with Ztalmy had a 31% median reduction in 28-day frequency of seizures compared to a median 7% reduction in the placebo arm. The FDA cautions that the drug can cause sleepiness and sedation, risks that increase if the drug is used with alcohol. The agency also noted that patients taking the drug should be monitored for suicidal behavior and thoughts.
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Dosing of Ztalmy is based on weight. According to a Marinus investor presentation, a patient who is four-and-a-half years old and weighs 16 kg (about 35.2 pounds) would have an average daily dose of 50 to 55 mg per kg. The wholesale cost of a bottle of the medicine is $2,425, making the average annual wholesale cost about $133,000 before any rebates or discounts. Marinus said in the investor presentation that it has committed to no price increases through the end of 2023.
Ganaxolone is a controlled substance, and the drug’s label cautions that use of the product may lead to abuse or dependence. The drug is subject to review by the Drug Enforcement Administration, which will schedule the drug according to the abuse and dependency risk. Marinus expects Ztalmy will launch in July following DEA scheduling.
CDKL5 deficiency is the first of several potential epilepsy indications for Ztalmy. A Phase 3 study is underway testing the drug in another rare, inherited form of epilepsy called tuberous sclerosis complex. The company is also developing a new formulation for use treating Lennox-Gastaut syndrome.
Marinus reported that its cash position as of the end of 2021 was $122.9 million. With the approval of Ztalmy, the FDA also awarded the company a priority review voucher. Marinus said it plans to sell that voucher; such vouchers have fetched $100 million or more.
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